NCT06925542

A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease

A Phase 1 Dose Evaluation Study of the Safety and Preliminary Efficacy of Anti-CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Adult Subjects With Refractory Autoimmune Disease

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 80 (estimated)
• Sponsor: CRISPR Therapeutics · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with refractory autoimmune diseases, including active systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM).

Detailed description

This study may enroll up to 80 subjects in total. CTX112 is a CD19 directed chimeric antigen receptor (CAR) T cell immunotherapy comprised of allogeneic T cells prepared for the treatment of refractory autoimmune diseases. The cells are from healthy adult volunteer donors that are genetically modified ex vivo using CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats/ CRISPR-associated protein 9) gene editing components (single guide RNA and Cas9 nuclease).

Conditions

• SLE (Systemic Lupus)
• Lupus Erythematosus, Systemic
• Lupus Nephritis
• Systemic Sclerosis
• Inflammatory Myopathy, Idiopathic
• Myositis
• Diffuse Cutaneous Systemic Sclerosis

Interventions

Type	Name	Description
BIOLOGICAL	CTX112	CTX112 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components)

Timeline

• Start date: 2025-03-10
• Primary completion: 2031-12-31
• Completion: 2031-12-31
• First posted: 2025-04-13
• Last updated: 2025-12-17

Locations

8 sites across 2 countries: United States, Germany

Regulatory

• FDA-regulated drug study